EP0707638A4 - REGULABLE NUCLEIC ACID FOR THERAPEUTIC USE AND METHODS OF USE THEREOF - Google Patents

REGULABLE NUCLEIC ACID FOR THERAPEUTIC USE AND METHODS OF USE THEREOF

Info

Publication number
EP0707638A4
EP0707638A4 EP94903456A EP94903456A EP0707638A4 EP 0707638 A4 EP0707638 A4 EP 0707638A4 EP 94903456 A EP94903456 A EP 94903456A EP 94903456 A EP94903456 A EP 94903456A EP 0707638 A4 EP0707638 A4 EP 0707638A4
Authority
EP
European Patent Office
Prior art keywords
ligand
ribozyme
target rna
molecules
cleaving
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Withdrawn
Application number
EP94903456A
Other languages
German (de)
French (fr)
Other versions
EP0707638A1 (en
Inventor
Shaji T George
Andy Shih
Jeffrey M Bockman
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sirna Therapeutics Inc
Original Assignee
Innovir Laboratories Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Innovir Laboratories Inc filed Critical Innovir Laboratories Inc
Publication of EP0707638A1 publication Critical patent/EP0707638A1/en
Publication of EP0707638A4 publication Critical patent/EP0707638A4/en
Withdrawn legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121—Hammerhead
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2730/00—Reverse transcribing DNA viruses
    • C12N2730/00011—Details
    • C12N2730/10011—Hepadnaviridae
    • C12N2730/10111—Orthohepadnavirus, e.g. hepatitis B virus
    • C12N2730/10122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Oncology (AREA)
  • Plant Pathology (AREA)
  • Communicable Diseases (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Saccharide Compounds (AREA)

Abstract

Construction of a regulatable ribozyme is described in which a ribozyme sequence is linked to a ligand-binding sequence, placing the activity of the ribozyme under the control of that ligand and requiring the presence of the ligand for activation or inactivation. RNA molecules are constructed in which one portion is capable of binding a ligand and the other portion is a ribozyme. After the selection of molecules which bind the ligand, a second selection process occurs in which the ligand-binding molecules are assayed for their catalytic function in the presence and absence of the ligand or 'co-drug.' In this manner regulatable ribozymes are selected for use in cleaving a target RNA in the presence of a ligand, or in cleaving a target RNA in the absence of a ligand. Thismethod and regulatable ribozymes are useful in cleaving a target RNA molecule in a controlled fashion. It is particularly useful when the target RNA molecule is present in a cell where it is not desirable to kill the host cell by complete inactivation of these RNA molecules.
EP94903456A 1992-12-04 1993-12-03 REGULABLE NUCLEIC ACID FOR THERAPEUTIC USE AND METHODS OF USE THEREOF Withdrawn EP0707638A4 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US98746592A 1992-12-04 1992-12-04
PCT/US1993/011783 WO1994013791A1 (en) 1992-12-04 1993-12-03 Regulatable nucleic acid therapeutic and methods of use thereof
US987465 1997-12-09

Publications (2)

Publication Number Publication Date
EP0707638A1 EP0707638A1 (en) 1996-04-24
EP0707638A4 true EP0707638A4 (en) 1998-05-20

Family

ID=25533286

Family Applications (1)

Application Number Title Priority Date Filing Date
EP94903456A Withdrawn EP0707638A4 (en) 1992-12-04 1993-12-03 REGULABLE NUCLEIC ACID FOR THERAPEUTIC USE AND METHODS OF USE THEREOF

Country Status (5)

Country Link
US (2) US5741679A (en)
EP (1) EP0707638A4 (en)
JP (3) JPH08507203A (en)
AU (1) AU679525B2 (en)
WO (1) WO1994013791A1 (en)

Families Citing this family (171)

* Cited by examiner, † Cited by third party
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US5683873A (en) * 1995-01-13 1997-11-04 Innovir Laboratories, Inc. EGS-mediated inactivation of target RNA
US5663064A (en) * 1995-01-13 1997-09-02 University Of Vermont Ribozymes with RNA protein binding site
US20040220128A1 (en) * 1995-10-26 2004-11-04 Sirna Therapeutics, Inc. Nucleic acid based modulation of female reproductive diseases and conditions
US20040142895A1 (en) * 1995-10-26 2004-07-22 Sirna Therapeutics, Inc. Nucleic acid-based modulation of gene expression in the vascular endothelial growth factor pathway
US7034009B2 (en) * 1995-10-26 2006-04-25 Sirna Therapeutics, Inc. Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
US20040102389A1 (en) * 1995-10-26 2004-05-27 Ribozyme Pharmaceuticals, Inc. Nucleic acid-mediated treatment of diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
US5877162A (en) * 1996-03-14 1999-03-02 Innovir Laboratories, Inc. Short external guide sequences
US20020106647A1 (en) * 1996-07-24 2002-08-08 Segal Andrew H. Nucleic acid compositions and methods of introducing nucleic acids into cells
NZ334492A (en) 1996-08-26 2000-09-29 Intelligene Ltd Catalytic nucleic acids (proto-nucleozyme), and their use for detecting agents by catalytic activity of proto-nucleozyme with a co-factor forming a nucleozyme(ribozyme)
WO1998027104A1 (en) * 1996-12-19 1998-06-25 Yale University Bioreactive allosteric polynucleotides
AU756301B2 (en) * 1997-08-20 2003-01-09 Somagenics, Inc. Antisense and antigene therapeutics with improved binding properties and methods for their use
JP4789294B2 (en) * 1997-08-22 2011-10-12 ソレクサ・インコーポレイテッド DNA extension and analysis using rolling primers
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US6248525B1 (en) 1998-03-30 2001-06-19 Yale University Method for identifying essential or functional genes
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US6995259B1 (en) * 1998-10-23 2006-02-07 Sirna Therapeutics, Inc. Method for the chemical synthesis of oligonucleotides
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US20040214255A1 (en) * 1999-04-22 2004-10-28 Myriad Genetics, Incorporated Compositions and methods for treating diabetes
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